275 Cost-effectiveness in Canada of azacitidine for the treatment of higher risk myelodysplastic syndromes and acute myeloid leukemia

Abstract

The open-label, prospective, observational study aimed to evaluate whether decitabine (DAC) plus thalidomide versus DAC monotherapy improved progression-free (PFS), overall survival (OS) and acute myeloid leukemia-free survival (AML-FS) for risk-tailored elderly patients with myelodysplastic syndromes (MDS).Enrolled 107 patients (age: 65–82 years) received DAC (52/107) or DAC plus thalidomide (55/107) therapy for MDS.35/52 patients (67.3%) with DAC therapy reached overall response (OR), compared with 36/55 patients (65.5%) in DAC + thalidomide regimen (P > 0.05). DAC + thalidomide administered did not gain more profits of PFS and OS than DAC monotherapy. Risk-tailored analysis showed that DAC + thalidomide therapy did not enhance PFS (48.9% versus 42.8%, P > 0.05) and OS (78.6% versus 71.2%, P > 0.05) when compared with simple DAC regimen. Nevertheless, DAC + thalidomide markedly improved OS over DAC monotherapy (50.6% versus 40.2%, P < 0.05) in high risk profile. Meanwhile, low risk group was superior to high risk group in AML-FS (57.2% versus 21.3%, P < 0.01), but DAC + thalidomide still did not prolong 2-year AML-FS when compared with DAC (32.4% versus 27.8%, P < 0.05). Moreover, thalidomide had a favorable toxicity profile as a single agent. In comparison with DAC monotherapy, the DAC + thalidomide regimen was relatively well tolerated. There was no severe non-hematological toxicity appearing in elderly patients with MDS.The study demonstrated that DAC + thalidomide improved 2-year OS for high risk patients. Thalidomide's proven activity and low toxicity profile made it an alternative treatment option for risk-tailored elderly patients with MDS.